– Study meets endpoints and demonstrates safety and tolerability of VXM01 –
VAXIMM AG, a Swiss-German biotech company focusing on oral cancer vaccines, announced today topline data from the first clinical trial of its investigational oral cancer vaccine VXM01. The randomized, placebo-controlled, double-blind Phase I/II dose escalation study met all key endpoints and demonstrated safety and tolerability.
The study code-named VXM01-01-DE enrolled 45 patients with inoperable pancreatic cancer at the Heidelberg University Hospital (Heidelberg, Germany). In addition to standard-of-care treatment, the patients received several doses of VXM01, a therapeutic vaccine targeting the tumor vasculature, or placebo.
The results of the study indicate that the vaccine was safe and well tolerated. No dose-limiting toxicities were observed. Besides this primary endpoint, several important secondary endpoints, including specific T-cell response and changes in tumor perfusion, were met. After vaccination with VXM01, a quarter of the patients showed a strongly increased T-cell mediated immune response against the target (VEGFR-2). This effect was distinct from fluctuations observed in the placebo-treated patients. Immunologically responding patients occurred already in the lowest dose group. A third of the VXM01-treated patients had a strong drop in tumor perfusion following the treatment, accompanied by corresponding changes in tumor-specific and angiogenesis-related biomarkers. Tumor perfusion changes in the treatment group were correlated with the VEGFR-2 specific effector and regulatory T-cell responses. More detailed results from the trial will be submitted for presentation at upcoming scientific meetings and for publication in a peer-reviewed journal.
“We are delighted to see that VXM01 was safe and well tolerated in the patients we treated,” said PD Dr. Hubertus Schmitz-Winnenthal, principal investigator of the study. “We are especially excited about the encouraging data observed in the two key secondary endpoints. The vaccine seems to be able to induce and enhance the VEGFR-2 specific T-cell response and to impact tumor perfusion in a good proportion of treated patients.”
“We are very encouraged by these data,” added Dr. Heinz Lubenau, General Manager of VAXIMM GmbH, a fully owned subsidiary of VAXIMM AG in Germany. “It provides a strong basis for continuing the development of VXM01 for the treatment of solid tumor diseases. Following regulatory approval, we plan to re-open the study VXM01-01-DE for further recruitment of pancreatic cancer patients.”
Dr. Klaus Breiner, Executive Chairman of VAXIMM AG commented: “We are very pleased with this outcome. This first-in-man study was already designed as a placebo-controlled trial, providing us with a high level of confidence in the validity of the results.”
OcellO B.V. and Merus B.V. have entered into a collaboration under which OcellO will provide screening services to Merus. OcellO, a biotech company providing drug discovery screening services using its proprietary high throughput 3D cell culture-based screening platform, will use its platform to profile bispecific antibodies designed by Merus for the treatment of cancer. Financial details of the collaboration have not been disclosed.
Mark Throsby, Chief Operating Officer at Merus, commented: “The strength of our approach is its ability to generate thousands of full-length, bispecific human IgG antibodies addressing combinations of targets expressed by tumor cells. OcellO is able to provide high quality data on the functional properties of large panels of bispecific antibodies. These data can’t be obtained from other in vitro screening assays and are pivotal in identifying antibody leads with unique modes of action.”
Merus B.V., a biopharmaceutical company focusing on innovative human antibody therapeutics, today announced that it has selected MCLA-117 as lead candidate for clinical studies in patients with acute myeloid leukemia (AML). MCLA-117 is a human, full-length IgG bispecific antibody, which activates the patient’s own immune system by simultaneously binding to the CLEC12A molecule expressed by AML tumor cells and the CD3 molecule expressed by T cells. MCLA-117-mediated co-engagement of CLEC12A and CD3 results in the potent killing of cancerous AML cells and their malignant precursors. Merus intends to start phase I clinical trials with MCLA-117 in 2014.
The antibody is based on Merus’ Biclonics™ ENGAGE platform. Human bispecific antibodies from this platform can be manufactured and administered like conventional, full-length IgG molecules, thereby providing for high yield, good stability and a long serum half life. In addition, bispecific antibodies from the Biclonics™ ENGAGE platform have a modified constant region to abrogate unwanted clinical cytokine release.
Prof. Gert Ossenkoppele from the department of hematology at the Free University Medical Centre in Amsterdam, The Netherlands, will be the lead investigator of the first MCLA-117 Phase I study.
Merus B.V., a biopharmaceutical company focused on innovative human antibody therapeutics, today announced the appointment of Setareh van Driel Shamsili, MD, Ph.D., as Chief Medical Officer (CMO).
Setareh van Driel Shamsili has more than 22 years of experience in general medicine, internal medicine and medical oncology. For the past 13 years, she has focused on both translational and clinical cancer research. She has more than eight years of experience in the pharmaceutical industry, including drug development, R&D, medical affairs, drug safety and pharmacovigilance and holds a PhD in Neuro-Oncology from Erasmus University Rotterdam.
Prior to joining Merus, Setareh van Driel Shamsili was Global Medical Leader Oncology at Astellas Pharma Global Development.